Featured Research

from universities, journals, and other organizations

Gene therapy shows promise as hemophilia treatment in animal studies

Date:
November 3, 2011
Source:
Wake Forest Baptist Medical Center
Summary:
For the first time, researchers have combined gene therapy and stem cell transplantation to successfully reverse the severe, crippling bleeding disorder hemophilia A in large animals, opening the door to the development of new therapies for human patients.

For the first time, researchers have combined gene therapy and stem cell transplantation to successfully reverse the severe, crippling bleeding disorder hemophilia A in large animals, opening the door to the development of new therapies for human patients.

Researchers at Wake Forest Baptist Medical Center's Institute for Regenerative Medicine, collaborating with other institutions, report in Experimental Hematology that a single injection of genetically-modified adult stem cells in two sheep converted the severe disorder to a milder form. The journal is a publication of the Society for Hematology and Stem Cells

"A new approach to treating severe hemophilia is desperately needed," said lead author Christopher D. Porada, Ph.D., associate professor of regenerative medicine at Wake Forest Baptist. "About 75 percent of the world doesn't have access to the current treatment -- therapy to replace missing clotting factors. This puts patients in most of the world at risk of severe and permanent disabilities."

Porada cautioned that challenges will need to be overcome before the treatment can be applied to humans, including that the sheep developed an immune response to the therapy that could decrease its effectiveness and duration.

There is currently no cure for the rare bleeding disorder hemophilia. People with this genetic disorder lack a protein, known as a clotting factor, needed for normal blood clotting. As a result, they may bleed for a longer time than others after an injury, as well as bleed internally, especially in joints such as the knees, ankles, and elbows. This bleeding can damage the organs and tissues and be life threatening. Even when life-threatening bleeds are prevented with replacement therapy, it doesn't prevent smaller bleeds within the joints that can cause pain and decreased mobility.

People with hemophilia A, the most common type, are missing clotting factor VIII. For the study, the researchers used a combined stem cell/gene therapy approach to increase levels of factor VIII produced by the animals.

The scientists first inserted a gene for factor VIII into engineered mesenchymal stem cells, a type of adult stem cell. The cells -- acting as a carrier for the gene -- were then injected into the abdominal cavity of the sheep. The scientists selected mesenchymal stem cells to carry the gene because they have the ability to migrate to sites of injury or inflammation.

In the treated animals, the cells migrated to the joints and stopped ongoing bleeding.In addition, all spontaneous bleeding events ceased, and the existing joint damage was completely reversed, restoring normal posture and gait to these crippled animals, and enabling them to resume a normal activity level.

However, a paradox of the treatment was that while the symptoms were eliminated, the sheep developed an immune response to factor VIII, suggesting that the treatment's effects would be reduced or shorter in duration. The scientists are currently working to learn why the immune response occurred and to develop strategies to prevent it.

"While preliminary, these findings could pave the way for a new therapy for hemophilia patients who experience debilitating bleeding in their joints," Porada said.

The research was supported by the National Institutes of Health.

Co-authors were Graηa Almeida-Porada (senior author) and Chung-Jung Kuo , both with Wake Forest Baptist; Chad Sanada, Evan Colletti, Esmail D. Zanjani, Walter Mandeville and John Hasenau, all with the University of Nevada at Reno; Robert Moot, Aflac Cancer Center and Blood Disorders Service; Christopher Doering, Emory Children's Center Pediatrics; and H. Trent Spencer, Emory University School of Medicine.


Story Source:

The above story is based on materials provided by Wake Forest Baptist Medical Center. Note: Materials may be edited for content and length.


Journal Reference:

  1. Christopher D. Porada, Chad Sanada, Chung-Jung Kuo, Evan Colletti, Walter Mandeville, John Hasenau, Esmail D. Zanjani, Robert Moot, Christopher Doering, H. Trent Spencer, Graηa Almeida-Porada. Phenotypic correction of hemophilia A in sheep by postnatal intraperitoneal transplantation of FVIII-expressing MSC. Experimental Hematology, 2011; DOI: 10.1016/j.exphem.2011.09.001

Cite This Page:

Wake Forest Baptist Medical Center. "Gene therapy shows promise as hemophilia treatment in animal studies." ScienceDaily. ScienceDaily, 3 November 2011. <www.sciencedaily.com/releases/2011/11/111103081434.htm>.
Wake Forest Baptist Medical Center. (2011, November 3). Gene therapy shows promise as hemophilia treatment in animal studies. ScienceDaily. Retrieved July 22, 2014 from www.sciencedaily.com/releases/2011/11/111103081434.htm
Wake Forest Baptist Medical Center. "Gene therapy shows promise as hemophilia treatment in animal studies." ScienceDaily. www.sciencedaily.com/releases/2011/11/111103081434.htm (accessed July 22, 2014).

Share This




More Health & Medicine News

Tuesday, July 22, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Gilead's $1000-a-Pill Drug Could Cure Hep C in HIV-Positive People

Gilead's $1000-a-Pill Drug Could Cure Hep C in HIV-Positive People

TheStreet (July 21, 2014) — New research shows Gilead Science's drug Sovaldi helps in curing hepatitis C in those who suffer from HIV. In a medical study, the combination of Gilead's Hep C drug with anti-viral drug Ribavirin cured 76% of HIV-positive patients suffering from the most common hepatitis C strain. Hepatitis C and related complications have been a top cause of death in HIV-positive patients. Typical medication used to treat the disease, including interferon proteins, tended to react badly with HIV drugs. However, Sovaldi's %1,000-a-pill price tag could limit the number of patients able to access the treatment. TheStreet's Keris Lahiff reports from New York. Video provided by TheStreet
Powered by NewsLook.com
$23.6 Billion Awarded To Widow In Smoking Lawsuit

$23.6 Billion Awarded To Widow In Smoking Lawsuit

Newsy (July 20, 2014) — Cynthia Robinson claims R.J. Reynolds Tobacco Company hid the health and addiction risks of its products, leading to the death of her husband in 1996. Video provided by Newsy
Powered by NewsLook.com
Tooth Plaque Provides Insight Into Diets Of Ancient People

Tooth Plaque Provides Insight Into Diets Of Ancient People

Newsy (July 19, 2014) — Research on plaque from ancient teeth shows that our prehistoric ancestor's had a detailed understanding of plants long before developing agriculture. Video provided by Newsy
Powered by NewsLook.com
Contaminated Water Kills 3 Babies in South African Town

Contaminated Water Kills 3 Babies in South African Town

AFP (July 18, 2014) — Contaminated water in South Africa's northwestern town of Bloemhof kills three babies and hospitalises over 500 people. The incident highlights growing fears over water safety in South Africa. Duration: 02:22 Video provided by AFP
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
 
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:  

Breaking News:
from the past week

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:  

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile iPhone Android Web
Follow Facebook Twitter Google+
Subscribe RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins